A Phase 1/2 interventional study of SGT-001 in Duchenne Muscular Dystrophy, sponsored by Solid Biosciences Inc.. Active, not recruiting at 2 sites in United States. Open to male participants aged 4 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-10-15.
Sponsored by Solid Biosciences Inc. · Phase 1/2, Interventional, and Treatment
This is a controlled, open-label, single-ascending dose study to evaluate the safety and tolerability of SGT-001 in adolescents and children with Duchenne muscular dystrophy (DMD). Participants will receive a single intravenous (IV) infusion of SGT-001 and will be followed for approximately 5 years.
The protocol was amended to drop the control arm after 4 participants were dosed.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 108 are open to participants now.
This study's enrollment of 12 is below the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Browse Muscular Dystrophy, Duchenne studies →Solid Biosciences Inc. is the lead sponsor of 6 studies on the registry; 4 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Additional inclusion/exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Single IV infusion of SGT-001 at starting dose
Genetic: SGT-001
Single IV infusion of SGT-001 at next ascending dose
Genetic: SGT-001
Untreated control group. After 1 year, treatment-eligible control participants will receive SGT-001 at the selected dose.
AAV9 vector containing muscle-specific promoter and microdystrophin construct
Number of Participants with Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Laboratory Parameters
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Vital Signs
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Physical Examinations
Time frame: Up to 5 years
Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG)
Time frame: Up to 5 years
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Western Blot (WB)
Time frame: Baseline, 12 months
Change from Baseline in Microdystrophin Protein Levels in Muscle Biopsies Using Immunofluorescence (IF)
Time frame: Baseline, 12 months
Change from Baseline in North Star Ambulatory Assessment (NSAA) score in Ambulatory Participants
Time frame: Baseline, 12 months
Change from Baseline in 6-minute walk test (6MWT) Distance in Ambulatory Participants
Time frame: Baseline, 12 months
Change from Baseline in Total Upper Limb Function, as Measured by the Total Performance of the Upper Limb (PUL) Functional Scale Score
Time frame: Baseline, 12 months
Change from Baseline in Respiratory Function, as Measured by Forced Vital Capacity (FVC) % Predicted, Forced Expiratory Volume in 1 second (FEV1) % Predicted, and Peak Expiratory Flow (PEF) % Predicted
Time frame: Baseline, 12 months
Change from Baseline in Ejection Fraction, As Measured by Echocardiography
Time frame: Baseline,12 months
Change from Baseline in Left Ventricular End Systolic Volume, As Measured by Echocardiography
Time frame: Baseline,12 months
Change from Baseline in Myocardial Peak Circumferential Strain (Ecc), As Measured by Echocardiography
Time frame: Baseline,12 months
Change from Baseline in Quality of Life as Measured by the Paediatric Quality of Life Inventory (PedsQL) Duchenne muscular dystrophy (DMD) module and self-reported outcome measures as measured by the PODCI DMD module
Time frame: Baseline, 12 months
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Muscular Dystrophy, Duchenne →
Solid Biosciences Inc.